Just under 300 children are affected out of a total of just over 7,000 patients of all ages in France. Until now, this treatment was reserved for people over 12 years old.
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A new hope for young patients. Kaftrio, a treatment that marks a great advance against cystic fibrosis, is now approved for some patients aged 6-11, health authorities announced on Tuesday March 29. Until now, the treatment was reserved in France for those over 12 years old,
This treatment will, through this procedure, immediately benefit from reimbursement by Social Security. However, Kaftrio will only benefit certain patients. These are those “who are heterozygous for the F508del mutation of the CFTR gene”, whose role is central in cystic fibrosis, explains the HAS. Just under 300 children are affected out of a total of just over 7,000 patients of all ages in France, estimates the HAS.
The treatment concerned consists of a combination of two drugs developed by the Vertex laboratory, Kalydeco and Kaftrio. It is Kaftrio which is considered a major advance against cystic fibrosis, a disease which causes abnormally thick mucus to secrete, obstructing the respiratory tract as well as the digestive system.
Most of the therapeutic approaches focus on the symptoms, via drugs or techniques such as physiotherapy, and have already made it possible to greatly extend life expectancy for a disease long considered fatal to children and adolescents.
But Kaftrio, which is part of an innovative class of drugs targeting the action of the protein at the origin of the disease, is considered by associations as a revolution which, for some patients, can transform cystic fibrosis into a chronic pathology. and stabilized.